Our Science & Approach

Zafgen has undertaken years of research to characterize the MetAP2 pathway and its effects in various bodily systems. We are focused on leveraging our proprietary understanding of this pathway to develop transformative therapeutics that treat the underlying biological mechanisms of a variety of metabolic diseases. Our approach has the potential to significantly improve the health and well-being of patients affected by a range of both rare and more prevalent metabolic disorders where MetAP2 plays a central role.

The Science & Promise of MetAP2 Inhibition

OUR PROGRAMS & PIPELINE

With our proprietary MetAP2 development platform, advanced chemistry insights and understanding of the unique characteristics of rare and more common complex metabolic disorders, Zafgen has developed several MetAP2 inhibitors, which modulate the cellular mechanisms that control the body’s ability to make and store fat and to use fat and glucose as an energy source.

The second-generation MetAP2 inhibitors now in our pipeline have each been optimized to preferentially target specific tissue systems relevant to the disease being pursued and to minimize exposure elsewhere in the body, which we anticipate will result in clinically differentiated efficacy and safety.

PRECLINICAL DEVELOPMENT

PHASE 1

PHASE 2

PHASE 3

ZGN-1061 for type 2 diabetes

PHASE 2

ZGN-1061 is being developed for patients with type 2 diabetes. We have reported positive full 12-week results for both cohorts of our Phase 2 proof-of-concept trial, including the 1.8 mg dose cohort of the trial.

The FDA has placed a clinical hold on the Investigational New Drug Application (IND) for the company’s first U.S. clinical trial of ZGN-1061.

ZGN-1258 for Prader-Willi syndrome

PRECLINICAL DEVELOPMENT

Zafgen has suspended plans to file an investigational new drug (IND) application for ZGN-1258, the Company’s candidate for rare metabolic disorders including Prader-Willi syndrome (PWS).

ZGN-1258 is designed to change the way the body metabolizes fat, reduce fat mass, and decrease hyperphagia. There are currently no approved medications to aid in controlling hyperphagia.

ZGN-1345

PRECLINICAL DEVELOPMENT

ZGN-1345, an orally dosed MetAP2 inhibitor specifically targeting the liver, has been formally advanced to development candidate stage as a differentiated third asset within the Company’s pipeline. Nonclinical models have shown positive preliminary results in multiple liver disease indications.